Short Bowel Syndrome

Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis is often the result of another diagnosis, or it may be due to trauma. The loss of the tissue causes the small intestine to struggle to absorb all of the fluids and nutrients it needs.

SBS patients often require long-term parenteral support. For adults with the disease, a treatment was approved in 2012 that helped reduce this dependence. However, until now, this same treatment was not available for children.

Thankfully, the FDA has just announced that they’ve extended their approval of the drug for children 1 year and older who require intravenous parenteral support. The drug is called teduglutide.

Teduglutide

Teduglutide is a GLP-2 analog which helps improve absorption within the intestines. With improved absorption, patients are able to reduce both the frequency of parenteral support, and also the volume.

Teduglutide is administered by injection. The recommended dose is the same for both adults and children at .05 mg/kg. This dose should be administered once each day.

Clinical Trials

A 24 week clinical trial supported this extended indication for teduglutide. 59 children with SBS who were between the ages of 1 and 17 participated in the study. Each patient had the choice of whether or not to take teduglutide or to receive the standard care for SBS. Those who chose to take the drug were randomly assigned to one of two doses (24 patients were given .025 mg/kg and 26 patients were given .05 mg/kg). Only 9 of the 59 participants chose not to receive teduglutide.

Results at 24 weeks for those taking a .05 mg/kg/day dose were as follows:

• 69% were able to reduce their volume of parenteral support by 20% or more
• Mean reduction of parenteral support volume was 42%
• 38% reduced the frequency of support by at least one day out of every week
• On average, infusion time was reduced by 3 hours each day
• 12% (3 participants) were able to completely stop infusions of parenteral support.

The safety profile of the treatment was similar between adults and pediatric patients. That said, there are some precautions and recommended tests prior to and during treatment. You can read more about these, and this study, here.

This newly approved indication will hopefully provide more children with SBS an improved quality of life while living with this rare condition.